Seeking Novel Leads in Rare Diseases and Neuroscience

Ipsen, a global biopharmaceutical company, is seeking novel and innovative leads to treat rare disease and rare neuroscience disease indications. A range of therapeutic areas and targets will be considered, but target diseases should be rare (>1.5 per million, <1 per 2,000). 

A range of mechanisms‑of‑action and classes of molecules are of interest, with a particular focus on small molecules, peptides, biologics, nucleic acids, RNA therapies (i.e. siRNAs) and antibody conjugates. Ipsen is open to a variety of drug delivery methods. 

Development Stage of Interest

Approaches of interest are preferred to be clinical‑stage assets at pre‑IND, clinical phase I or clinical phase II. Research should be validated empirically, e.g. through in vitro animal proof of concept studies. 

Target Clinical Indications

The main areas of focus can be related to: endocrinology, hepatic disease, inborn errors of metabolism, neuromuscular disease and musculoskeletal disease. 

A full list of diseases of interest can be accessed here.

Out of Scope

• Ultra‑rare diseases (<1.5 per million)
• Assets that treat rare dermatology, pulmonary, haematology, ophthalmology and infectious indications only 
• Neurological diseases such as Alzheimer’s, Parkinson’s, multiple sclerosis, general pain and psychiatry
• Gene therapy, cell therapy, stem cell/regenerative therapies, vaccines, biosimilars/generic, traditional or herbal medicine, natural products, and medical devices
• Research‑stage assets (unless clinic‑ready or with empirical validation)

Submission Information

Submission of one-page, 200-300 word briefs are encouraged, along with any optional supplementary information e.g. relevant publications. Intellectual property status of the research should be included where relevant. Applicants are encouraged to fill in an application form to provide more in-depth information alongside their submission to this campaign. In submitting to this campaign, you confirm that your submission contains only non-confidential information.

Ipsen are seeking a variety of collaboration opportunities including asset acquisition and partnering, to be decided on a case-by-case basis.